AMT-130 (ifezuntirgene inilparvovec) for Huntington's disease
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The FDA is expected to decide on AMT-130 for Huntington's disease in an undisclosed window, under a BLA from uniQure.
What is being decided
uniQure has a BLA under FDA review for AMT-130 (ifezuntirgene inilparvovec) in Huntington's disease. One-time gene therapy delivered by neurosurgery into the striatum to silence the huntingtin gene; would be the first treatment shown to slow Huntington's disease. uniQure submitted the BLA for accelerated approval on September 2, 2026 and requested priority review; the FDA's filing decision is due within 60 days and no target action date has been disclosed. Holds Breakthrough Therapy, RMAT and Fast Track designations.
Who this decision matters to
Huntington disease is an autosomal dominant neurodegenerative disorder caused by an expanded CAG trinucleotide repeat in the huntingtin gene. The progressive disease causes movement problems, cognitive decline, and psychiatric symptoms. Adult-onset disease typically appears ages 30-50, with juvenile-onset forms presenting earlier with different symptom profiles. Multiple disease-modifying therapies are in clinical trials.
Prevalence: 5-10 per 100,000 people of European descent; lower in other populations. See our full Huntington Disease page for current treatments, recruiting trials, and community resources.
What each outcome would mean
An approval starts a second race rather than ending the first one: specialty pharmacy setup, insurance review, and patient assistance typically take weeks even when everything goes right. Our guide to the 90 days after a rare disease approval explains the timeline and the moves families can make on day 1.
A complete response letter would mean the FDA declined to approve in the application's current form. CRLs are often about manufacturing or data presentation rather than efficacy, and resubmission is common. Either way, this page updates with the outcome and what it means.
Where Huntington Disease treatment stands today
Read our full Huntington Disease treatment summary
Huntington disease research is advancing 3 distinct approaches to lower mutant huntingtin levels. Antisense oligonucleotides (ASOs) like tominersen reduce HTT mRNA through intrathecal injection.
Gene therapies like AMT-130 use a viral vector to deliver a one-time genetic instruction to the brain that lowers production of the huntingtin protein. Splicing modifiers like votoplam (PTC518) are oral drugs that alter HTT pre-mRNA splicing.
Each approach has different trial designs and eligibility requirements. Genetic testing to confirm your CAG repeat length is the first step for any HD trial.
Premanifest gene carriers (CAG 40+ with no motor symptoms) have a growing number of prevention trials aimed at delaying or preventing symptom onset. If you take fluoxetine, paroxetine, or bupropion for depression, check trial eligibility carefully, as these are strong CYP2D6 inhibitors excluded from some trials.
Do not change an antidepressant on your own to qualify for a trial, and ask your neurologist whether another option is safe for you. VMAT2 inhibitors for chorea are generally allowed at a stable dose.
The Huntington's Disease Society of America provides resources and genetic counseling referrals.
The Huntington Disease page has every treatment and trial →Meanwhile, 41 Huntington Disease trials are recruiting
Whatever the FDA decides here, research on Huntington Disease does not stop. A few currently enrolling studies, US sites first:
We'll email you when the decision is public, with what it means in plain language, and when new trials open for Huntington Disease. Unsubscribe anytime.
Frequently asked questions
When will the FDA decide on AMT-130?
uniQure has not disclosed a decision date.
Who disclosed it: uniQure. The FDA does not publish these dates.
Can it move: the FDA can act early, and a major amendment to the application can push the date back 3 months.
What is AMT-130 being reviewed for?
uniQure submitted a BLA for AMT-130 in Huntington's disease.
What to know: One-time gene therapy delivered by neurosurgery into the striatum to silence the huntingtin gene; would be the first treatment shown to slow Huntington's disease. uniQure submitted the BLA for accelerated approval on September 2, 2026 and requested priority review; the FDA's filing decision is due within 60 days and no target action date has been disclosed. Holds Breakthrough Therapy, RMAT and Fast Track designations.
Is there already an FDA-approved treatment for Huntington Disease?
Yes. Trial Friend's drug list includes 3 FDA-approved drugs for Huntington Disease.
Approved: Xenazine (tetrabenazine), Austedo (deutetrabenazine), Ingrezza (valbenazine).
Check: the Huntington Disease page also searches FDA drug labels for approved treatments.
Can I get AMT-130 before the FDA decides?
Usually only through a clinical trial or an expanded access program, and the company decides whether to offer one.
Trials: 41 Huntington Disease trials are recruiting now. See them.
Check your fit: Match Me compares your details with every open trial.
Expanded access: your doctor can ask uniQure directly. Registered programs are listed on ClinicalTrials.gov.
How much will AMT-130 cost?
uniQure has not announced a price. Drugmakers usually set it around approval.
Help paying: drugmaker programs and charity funds usually open at launch.
Find help: the patient assistance finder lists programs by insurance type.
What happens after the AMT-130 decision?
This page updates with the outcome the day it is announced.
If approved: getting the drug still takes time. Specialty pharmacy setup, insurance review and patient assistance often take weeks. Our guide covers what families can do on day 1.
If not approved: a complete response letter means the FDA declined the application in its current form. Many are about manufacturing or how data were presented, and companies often resubmit.
Where this date comes from
The FDA does not publish PDUFA dates; companies disclose them. This one comes from uniQure press release, Sep 2 2026. Dates can move, and the FDA can act early or late. This page rechecks against our calendar, which is re-verified daily.